drug · oligonucleotide
WAINUA (AUTOINJECTOR)
updated 3mo agoby $AZN ASTRAZENECA PLC🔗 ChEMBL
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top 2Oligonucleotide drug with a maximum clinical stage of Approval (across all indications), with an approval for familial amyloid neuropathy and polyneuropathy and 1 investigational indication.
targets
Transthyretin mRNA
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nothing on the calendar yet
News(10)
- BridgeBio continues ATTR ascent as Alnylam’s Amvuttra falls short of expectationsBridgeBio Pharma’s transthyretin amyloidosis medicine Attruby is off to a strong launch, while Alnylam lowered full-year sales guidance for competitor Amvuttra.biospace · 3h ago
- Intellia finds genetic suspect for liver safety signals with ATTR gene therapyIn November 2025, one patient died after receiving Intellia Therapeutics’ investigational gene silencer for transthyretin amyloidosis.biospace · 5d ago
- ‘Unlikely’ AstraZeneca-BMS mega-merger would be largest pharma deal everIf AstraZeneca and Bristol Myers Squibb were to successfully become one company, analysts say it could reset the current deal environment, which has picked up greatly in the first half of the year.biospace · 9d ago
- 5 companies advancing ATTR assets in the wake of Wainua’s failThe Phase 3 failure of AstraZeneca and Ionis Pharmaceuticals’ antisense therapy in transthyretin amyloid cardiomyopathy last month left the space reeling—and readjusting. BioSpace looks at five con…biospace · 9d ago
- Alnylam absorbs $10.8B blowback after cutting full-year guidance for TTR franchiseAlnylam’s revenue cut “may create a near term credibility overhang and could “raise questions on peak sales potential, even if there’s tailwinds in the competitive landscape,” according to Stifel.biospace · 12d ago
- Capricor and Replimune prep for FDA adcomms, Q2 earnings roll on, Sarepta’s new leader, moreThe FDA published briefing documents ahead of advisory committee meetings for therapies from Capricor Therapeutics and Replimune indicate an uphill battle for the biotechs; GSK and AstraZeneca outl…biospace · 14d ago
- AstraZeneca has no plans to ramp up dealmaking in wake of Wainua failureAstraZeneca remains "very much on track" to hit its 2030 goal of reaching $80 billion in revenue despite the recent late-stage failure of Wainua in ATTR-cardiomyopathy, CEO Pascal Soriot said durin…biospace · 16d ago
- Q2 earnings underway, FDA’s CRL kerfuffle, IPOs full speed ahead in H2Second quarter earnings continue to roll with Novartis and Roche reporting this week; Eli Lilly’s AtaiBeckley buy for up to $3.8 billion offers clear “validation” for the fledgling psychedelics spa…biospace · 21d ago
- Royalty Pharma pens $425M deal for slice of sales on AstraZeneca’s ATTR drugRoyalty Pharma has secured a stake in another transthyretin-mediated amyloidosis drug, this time paying $125 million upfront to Neurimmune for a cut of the sales of its AstraZeneca-partnered med.fiercebiotech · 21d ago
- Wainua flop narrows AstraZeneca’s path to $80B goal ahead of high-risk readoutsWith a Phase 3 failure denting the rare disease drug’s prospects, AstraZeneca has little room for error as it plots a path toward its ambitious 2030 sales target.biospace · 21d ago
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